Clinical Trials in Poland / NCT05392114
Active, not recruiting
Phase 3
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
NCT05392114 · tracked via the Priya Life Science Poland tracker
Condition(s) studied
Hereditary Angioedema
Investigational drug(s) / intervention(s)
Donidalorsen: Donidalorsen will be administered by SC injection.
Study summary
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
Eligibility
Inclusion Criteria
1. Participants and, as applicable, legally authorized representatives (i.e., parent(s)/legal guardian), must provide written and signed informed consent form (ICF).
2. Participants must have access to, and the ability to use, ≥ 1 acute medication(s) (e.g., plasma-derived or recombinant C1-INH concentrate or a bradykinin receptor (BK) 2-receptor antagonist) to treat angioedema attacks.
Open-Label Extension Participants ONLY:
3. Satisfactory completion of ISIS 721744-CS5 (randomized placebo-controlled index study) through Week 25 or participants who are allowed to exit ISIS 721744-CS5 study per protocol with an acceptable safety and tolerability profile.
New (not previously on donidalorsen) Participants ONLY:
4. Participants must be aged ≥ 12 years at the time of informed consent and, as applicable, assent.
5. Participants must have a documented diagnosis of HAE-1/HAE-2.
6. Participants must be on a stable dose (≥ 12 weeks) of prophylaxis treatment with lanadelumab or berotralstat or SC C1-esterase inhibitor prior to the Screening Period.
Exclusion Criteria
Open-Label Extension Participants:
1. Have any new condition or worsening of an existing condition or change or anticipated change in medication.
New (not previously on donidalorsen) Participants ONLY:
2. Concurrent diagnosis of any other type of recurrent angioedema, including acquired, idiopathic angioedema or HAE with normal C1-INH (also known as HAE Type III).
3. Anticipated change in the use of concurrent androgen or tranexamic acid prophylaxis used to prevent angioedema attacks.
4. Any clinically-significant abnormalities in screening laboratory values.
5. Malignancy within 5 years of Screening, except for non-melanoma skin cancers, cervical in situ carcinoma, breast ductal carcinoma in situ, or stage 1 prostate carcinoma that has been successfully treated.
6. Hypersensitivity to the active substance (donidalorsen) or to any of the excipients.
7. Treatment with another investigational drug (non-oligonucleotide) or biological agent within 1 month of Screening or 5 half-lives of investigational agent, whichever is longer.
8. Recent history of, or current drug or alcohol abuse.
9. Participated in a prior donidalorsen study.
10. Exposure to any of the following medications:
1. Angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption.
2. Oligonucleotides (including small interfering ribonucleic acid \[siRNA\]) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses received. This exclusion does not apply to vaccines.
Primary outcome measure(s)
- Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity — Up to approximately 70 weeks, plus 104 weeks for Group 1; up to approximately 76 weeks, plus 104 weeks for Group 2
Trial sites (50)
| Facility | City | Region | Status |
|---|---|---|---|
| Ionis Investigative Site | Paradise Valley | Arizona | |
| Ionis Investigative Site | La Jolla | California | |
| Ionis Investigative Site | Santa Monica | California | |
| Ionis Investigative Site | Walnut Creek | California | |
| Ionis Investigative Site | Tampa | Florida | |
| Ionis Investigative Site | Kansas City | Kansas | |
| Ionis Investigative Site | Boston | Massachusetts | |
| Ionis Investigative Site | Ann Arbor | Michigan | |
| Ionis Investigative Site | Detroit | Michigan | |
| Ionis Investigative Site | St Louis | Missouri | |
| Ionis Investigative Site | Mooresville | North Carolina | |
| Ionis Investigative Site | Cincinnati | Ohio | |
| Ionis Investigative Site | Columbus | Ohio | |
| Ionis Investigative Site | Hershey | Pennsylvania | |
| Ionis Investigative Site | Dallas | Texas | |
| Ionis Investigative Site | Murray | Utah | |
| Ionis Investigative Site | Edegem | Belgium | |
| Ionis Investigative Site | Sofia | Bulgaria | |
| Ionis Investigative Site | Sofia | Bulgaria | |
| Ionis Investigative Site | Ottawa | Ontario | |
| Ionis Investigative Site | Edmonton | Canada | |
| Ionis Investigative Site | La Tronche | France | |
| Ionis Investigative Site | Marseille | France | |
| Ionis Investigative Site | Paris | France | |
| Ionis Investigative Site | Berlin | Germany | |
| Ionis Investigative Site | Frankfurt | Germany | |
| Ionis Investigative Site | München | Germany | |
| Ionis Investigative Site | Ashkelon | Israel | |
| Ionis Investigative Site | Haifa | Israel | |
| Ionis Investigative Site | Tel Aviv | Israel | |
| Ionis Investigative Site | Catania | Italy | |
| Ionis Investigative Site | Naples | Italy | |
| Ionis Investigative Site | Padova | Italy | |
| Ionis Investigative Site | Palermo | Italy | |
| Ionis Investigative Site | San Donato Milanese | Italy | |
| Ionis Investigative Site | Amsterdam | Netherlands | |
| Ionis Investigative Site | Groningen | Netherlands | |
| Ionis Investigative Site | Krakow | Poland | |
| Ionis Investigative Site | San Juan | Puerto Rico | |
| Ionis Investigative Site | Barcelona | Spain |
+ 10 more sites — see the full list on the official registry below.
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REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
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Other trials for the same condition
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
Enrolling by invitation
Berotralstat Treatment in Children With Hereditary Angioedema
Active, not recruiting
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Enrolling by invitation
STOP-HAE: A Phase 3 Study of ADX-324 in HAE
Recruiting
A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema
Active, not recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05392114 on ClinicalTrials.gov ↗ ← All trials in Poland